CA2796722C - Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types - Google Patents
Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types Download PDFInfo
- Publication number
- CA2796722C CA2796722C CA2796722A CA2796722A CA2796722C CA 2796722 C CA2796722 C CA 2796722C CA 2796722 A CA2796722 A CA 2796722A CA 2796722 A CA2796722 A CA 2796722A CA 2796722 C CA2796722 C CA 2796722C
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- nucleic acid
- compositions
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- oligonucleotide molecules
- selective delivery
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/0002—General or multifunctional contrast agents, e.g. chelated agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/4525—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
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- A61K31/47—Quinolines; Isoquinolines
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- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
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- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
- C07D207/46—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with hetero atoms directly attached to the ring nitrogen atom
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D217/00—Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems
- C07D217/02—Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines
- C07D217/04—Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines with hydrocarbon or substituted hydrocarbon radicals attached to the ring nitrogen atom
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2320/00—Applications; Uses
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- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32551510P | 2010-04-19 | 2010-04-19 | |
| EP10382087.4 | 2010-04-19 | ||
| US61/325,515 | 2010-04-19 | ||
| EP10382087A EP2380595A1 (en) | 2010-04-19 | 2010-04-19 | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| EP11382031 | 2011-02-09 | ||
| EP11382031.0 | 2011-02-09 | ||
| PCT/EP2011/056270 WO2011131693A2 (en) | 2010-04-19 | 2011-04-19 | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2796722A1 CA2796722A1 (en) | 2011-10-27 |
| CA2796722C true CA2796722C (en) | 2021-06-01 |
Family
ID=44063382
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2796722A Active CA2796722C (en) | 2010-04-19 | 2011-04-19 | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US9193969B2 (enExample) |
| EP (2) | EP2560687B1 (enExample) |
| JP (1) | JP5819401B2 (enExample) |
| KR (1) | KR20130103662A (enExample) |
| AR (1) | AR083715A1 (enExample) |
| AU (4) | AU2011244321A1 (enExample) |
| BR (1) | BR112012026471A2 (enExample) |
| CA (1) | CA2796722C (enExample) |
| DK (1) | DK2560687T3 (enExample) |
| ES (1) | ES2638309T3 (enExample) |
| LT (1) | LT2560687T (enExample) |
| MX (1) | MX342764B (enExample) |
| RU (1) | RU2664452C2 (enExample) |
| WO (1) | WO2011131693A2 (enExample) |
Families Citing this family (56)
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| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
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| EA202091012A1 (ru) | 2017-10-25 | 2020-08-27 | Янссен Фармасьютикалз, Инк. | Композиции фосфорилированных тау-пептидов и их применения |
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| MA51634A (fr) | 2018-01-12 | 2020-11-18 | Roche Innovation Ct Copenhagen As | Oligonucléotides antisens d'alpha-synucléine et leurs utilisations |
| US12241065B2 (en) | 2018-01-12 | 2025-03-04 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| CN120795059A (zh) | 2018-08-27 | 2025-10-17 | 瑞泽恩制药公司 | 拉曼光谱在下游纯化中的应用 |
| ES2698565B2 (es) * | 2018-12-12 | 2019-07-16 | Univ Madrid Complutense | Procedimiento para la elaboración de nanopartículas lipídicas, y nanopartículas lipídicas con los macrófagos cerebrales como células diana |
| CN113710269A (zh) | 2019-02-08 | 2021-11-26 | Ac免疫有限公司 | 磷酸化Tau肽疫苗的安全施用方法 |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| EP3952919A2 (en) | 2019-04-11 | 2022-02-16 | Northeastern University | Oligonucleotide-polymer miktoarm conjugates and methods of use |
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| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| EP4277635A4 (en) * | 2021-01-18 | 2024-12-25 | Yeda Research and Development Co. Ltd | MODIFIED MIR-135, CONJUGATED FORM THEREOF, AND USES THEREOF |
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| US12396981B2 (en) | 2023-03-09 | 2025-08-26 | William Shulman | Methods of using DMT |
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| US20040242594A1 (en) * | 2001-11-17 | 2004-12-02 | Dan Peters | Prodrugs of antidepressants and their use for treating depressions |
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| JP2007525211A (ja) * | 2004-01-19 | 2007-09-06 | テクニオン リサーチ アンド ディベロップメント ファウンデーション リミテッド | パーキンソン病の診断検査 |
| WO2007050789A2 (en) * | 2005-10-25 | 2007-05-03 | Nëdken Corporation | Glycosyl-oligonucleotide conjugates and methods |
| GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| WO2008033285A2 (en) * | 2006-09-15 | 2008-03-20 | The Trustees Of Culumbia University In The City Of New York | Delivery of double-stranded rna into the central nervous system |
| MX2010006925A (es) * | 2007-12-20 | 2011-05-02 | Angiochem Inc | Conjugados de polipeptido-acido nucleico y sus usos. |
| GB0809476D0 (en) * | 2008-05-23 | 2008-07-02 | Cambridge Entpr Ltd | Treatment of neurodegenerative disorders |
| DE102009058769A1 (de) * | 2009-12-16 | 2011-06-22 | MagForce Nanotechnologies AG, 10589 | Temperaturabhängige Aktivierung von katalytischen Nukleinsäuren zur kontrollierten Wirkstofffreisetzung |
| LT2560687T (lt) | 2010-04-19 | 2017-09-25 | Nlife Therapeutics S.L. | Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams |
| WO2012068405A2 (en) * | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| ES2967530T3 (es) | 2012-10-26 | 2024-04-30 | Palomo Ltd | Composiciones y métodos para el tratamiento de la enfermedad de Parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| EP2911697A1 (en) | 2012-10-26 | 2015-09-02 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to cell types |
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| JP2013525330A (ja) | 2013-06-20 |
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| AU2019203887A1 (en) | 2019-07-18 |
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| EP3231446B1 (en) | 2026-01-07 |
| CN103025357A (zh) | 2013-04-03 |
| AU2011244321A1 (en) | 2012-11-15 |
| AU2019203887B2 (en) | 2020-01-23 |
| AU2020202533B2 (en) | 2022-01-20 |
| US9193969B2 (en) | 2015-11-24 |
| LT2560687T (lt) | 2017-09-25 |
| WO2011131693A3 (en) | 2012-02-16 |
| BR112012026471A2 (pt) | 2016-11-29 |
| JP5819401B2 (ja) | 2015-11-24 |
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| KR20130103662A (ko) | 2013-09-24 |
| EP3231446A1 (en) | 2017-10-18 |
| EP2560687A2 (en) | 2013-02-27 |
| RU2664452C2 (ru) | 2018-08-17 |
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